RyCarma Therapeutics

About RyCarma Therapeutics

Originally established in 2004 as ARMGO Pharma, the company rebranded to RyCarma Therapeutics in January 2025, signaling a renewed strategic direction. The company was founded by Dr. Andrew R. Marks, a distinguished scientist from Columbia University, based on his groundbreaking research on ryanodine receptors (RyRs). Dr. Marks, a cardiologist and professor, has an extensive background in molecular cardiology and cellular biophysics, and his work led to the development of the first drug-eluting stents approved by the FDA in 2003. His research into 'leaky' intracellular calcium release channels and their role in various diseases forms the scientific foundation of the company.

RyCarma Therapeutics operates as a clinical-stage biopharmaceutical company focused on developing a novel class of small-molecule drugs known as Rycals®. The business model centers on the discovery, development, and commercialization of these proprietary therapeutics for debilitating cardiac, musculoskeletal, and neurological disorders. The company holds an exclusive worldwide license from Columbia University for its RyR technology. Its revenue generation will be contingent on the successful clinical development and eventual marketing of its drug candidates. The primary clients are patients suffering from diseases caused by dysfunctional RyR channels. The company targets both rare diseases and more common conditions, such as heart failure.

The company's core product is a pipeline of Rycals®, which are oral agents designed to repair leaky RyR calcium channels. These leaks disrupt normal muscle function and contribute to the progression of numerous diseases. The lead drug candidate, surlorian (also known as ARM210 or S48168), is an allosteric modulator that preferentially binds to and stabilizes the closed state of leaky RyR channels, thereby restoring normal calcium flow. This mechanism has the potential to be a disease-modifying treatment rather than merely addressing symptoms. Surlorian is currently advancing into Phase 2 clinical trials for two primary indications: heart failure with reduced ejection fraction (HFrEF) and Ryanodine Receptor 1-Related Myopathies (RYR1-RM), a rare and severe muscle disease. A recently concluded Phase 1b trial for RYR1-RM showed that ARM210 was well-tolerated and demonstrated early signs of clinical efficacy, including reduced fatigue and improved muscle strength in patients. The drug has been granted orphan drug designation by the FDA for both RYR1-RM and Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT).